WO2003102131A3 - Nucleic acid mediated disruption of hiv fusogenic peptide interactions - Google Patents

Nucleic acid mediated disruption of hiv fusogenic peptide interactions Download PDF

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Publication number
WO2003102131A3
WO2003102131A3 PCT/US2003/012626 US0312626W WO03102131A3 WO 2003102131 A3 WO2003102131 A3 WO 2003102131A3 US 0312626 W US0312626 W US 0312626W WO 03102131 A3 WO03102131 A3 WO 03102131A3
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WO
WIPO (PCT)
Prior art keywords
hiv
nucleic acid
fusogenic peptide
acid mediated
peptide interactions
Prior art date
Application number
PCT/US2003/012626
Other languages
French (fr)
Other versions
WO2003102131A2 (en
Inventor
Dennis Macejak
Lawrence Blatt
James Mcswiggen
Original Assignee
Sirna Therapeutics Inc
Dennis Macejak
Lawrence Blatt
James Mcswiggen
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Priority claimed from US10/157,580 external-priority patent/US20030124513A1/en
Priority claimed from US10/225,023 external-priority patent/US20030175950A1/en
Application filed by Sirna Therapeutics Inc, Dennis Macejak, Lawrence Blatt, James Mcswiggen filed Critical Sirna Therapeutics Inc
Priority to AU2003228667A priority Critical patent/AU2003228667A1/en
Publication of WO2003102131A2 publication Critical patent/WO2003102131A2/en
Publication of WO2003102131A3 publication Critical patent/WO2003102131A3/en
Priority to US10/892,922 priority patent/US20050124569A1/en
Priority to US10/923,473 priority patent/US20050191618A1/en
Priority to US12/334,181 priority patent/US20090264504A1/en
Priority to US12/777,767 priority patent/US8008472B2/en

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/115—Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1131—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against viruses
    • C12N15/1132—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against viruses against retroviridae, e.g. HIV
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/121—Hammerhead
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/332—Abasic residue
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed

Abstract

The present invention relates to nucleic acid aptamers that bind to HIV envelope glycoprotein, gp120 and/or gp41 and methods for their use alone or in combination with other therapies, such as HIV RT inhibitors and HIV protease inhibitors. Also disclosed are nucleic acids such as siRNA, antisense, and enzymatic nucleic acid molecules that can modulate the expression of HIV env genes and HIV viral replication. The compounds and methods of the invention are expected to inhibit HIV viral fusion, cell entry, gene expression, and replication.
PCT/US2003/012626 2001-05-18 2003-04-22 Nucleic acid mediated disruption of hiv fusogenic peptide interactions WO2003102131A2 (en)

Priority Applications (5)

Application Number Priority Date Filing Date Title
AU2003228667A AU2003228667A1 (en) 2002-04-22 2003-04-22 Nucleic acid mediated disruption of hiv fusogenic peptide interactions
US10/892,922 US20050124569A1 (en) 2001-05-18 2004-07-16 RNA interference mediated inhibition of CXCR4 gene expression using short interfering nucleic acid (siNA)
US10/923,473 US20050191618A1 (en) 2001-05-18 2004-08-20 RNA interference mediated inhibition of human immunodeficiency virus (HIV) gene expression using short interfering nucleic acid (siNA)
US12/334,181 US20090264504A1 (en) 2001-05-18 2008-12-12 RNA INTERFERENCE MEDIATED INHIBITION OF HUMAN IMMUNODEFICIENCY VIRUS (HIV) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
US12/777,767 US8008472B2 (en) 2001-05-29 2010-05-11 RNA interference mediated inhibition of human immunodeficiency virus (HIV) gene expression using short interfering nucleic acid (siNA)

Applications Claiming Priority (20)

Application Number Priority Date Filing Date Title
US37472202P 2002-04-22 2002-04-22
US60/374,722 2002-04-23
US10/157,580 2002-05-29
US10/157,580 US20030124513A1 (en) 2001-05-29 2002-05-29 Enzymatic nucleic acid treatment of diseases or conditions related to levels of HIV
US38678202P 2002-06-06 2002-06-06
US60/386,782 2002-06-06
US39803602P 2002-07-23 2002-07-23
US60/398,036 2002-07-23
US10/225,023 US20030175950A1 (en) 2001-05-29 2002-08-21 RNA interference mediated inhibition of HIV gene expression using short interfering RNA
US10/225,023 2002-08-21
US40678402P 2002-08-29 2002-08-29
US60/406,784 2002-08-29
US40837802P 2002-09-05 2002-09-05
US60/408,378 2002-09-05
US40929302P 2002-09-09 2002-09-09
US60/409,293 2002-09-09
US44012903P 2003-01-15 2003-01-15
US60/440,129 2003-01-15
PCT/US2003/005190 WO2003070193A2 (en) 2002-02-20 2003-02-20 RNA INTERFERENCE MEDIATED INHIBITION OF HIV GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
USPCT/US03/05190 2003-02-20

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
US10/420,194 Continuation-In-Part US20040006035A1 (en) 2001-05-18 2003-04-22 Nucleic acid mediated disruption of HIV fusogenic peptide interactions

Related Child Applications (2)

Application Number Title Priority Date Filing Date
US10/892,922 Continuation-In-Part US20050124569A1 (en) 2001-05-18 2004-07-16 RNA interference mediated inhibition of CXCR4 gene expression using short interfering nucleic acid (siNA)
US10/923,473 Continuation-In-Part US20050191618A1 (en) 2001-05-18 2004-08-20 RNA interference mediated inhibition of human immunodeficiency virus (HIV) gene expression using short interfering nucleic acid (siNA)

Publications (2)

Publication Number Publication Date
WO2003102131A2 WO2003102131A2 (en) 2003-12-11
WO2003102131A3 true WO2003102131A3 (en) 2004-04-01

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2003/012626 WO2003102131A2 (en) 2001-05-18 2003-04-22 Nucleic acid mediated disruption of hiv fusogenic peptide interactions

Country Status (2)

Country Link
AU (1) AU2003228667A1 (en)
WO (1) WO2003102131A2 (en)

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8957198B2 (en) 2003-02-03 2015-02-17 Medtronic, Inc. Compositions, devices and methods for treatment of Huntington's disease through intracranial delivery of sirna
US9133517B2 (en) 2005-06-28 2015-09-15 Medtronics, Inc. Methods and sequences to preferentially suppress expression of mutated huntingtin
US9273356B2 (en) 2006-05-24 2016-03-01 Medtronic, Inc. Methods and kits for linking polymorphic sequences to expanded repeat mutations
US9375440B2 (en) 2006-11-03 2016-06-28 Medtronic, Inc. Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB0226374D0 (en) * 2002-11-12 2002-12-18 Isis Innovation Ligands
US7618948B2 (en) 2002-11-26 2009-11-17 Medtronic, Inc. Devices, systems and methods for improving and/or cognitive function through brain delivery of siRNA
EP1993584B1 (en) * 2006-02-02 2012-05-30 Allergan, Inc. Inhibitors of CXCR4 activity for use in the treatment of ocular disorders
CA2812962C (en) 2010-09-22 2020-03-31 Alios Biopharma, Inc. Azido nucleosides and nucleotide analogs
CN106086075B (en) * 2016-06-23 2019-08-06 福建医科大学 The construction method of CXCR4RNAi slow virus carrier

Citations (3)

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Publication number Priority date Publication date Assignee Title
WO1994001550A1 (en) * 1992-07-02 1994-01-20 Hybridon, Inc. Self-stabilized oligonucleotides as therapeutic agents
WO2000001846A2 (en) * 1998-07-03 2000-01-13 Devgen N.V. Characterisation of gene function using double stranded rna inhibition
CA2359180A1 (en) * 1999-01-30 2000-08-03 Roland Kreutzer Method and medicament for inhibiting the expression of a given gene

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1994001550A1 (en) * 1992-07-02 1994-01-20 Hybridon, Inc. Self-stabilized oligonucleotides as therapeutic agents
WO2000001846A2 (en) * 1998-07-03 2000-01-13 Devgen N.V. Characterisation of gene function using double stranded rna inhibition
CA2359180A1 (en) * 1999-01-30 2000-08-03 Roland Kreutzer Method and medicament for inhibiting the expression of a given gene

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
PARK ET AL.: "Specific inhibition of HIV-1 gene expression by double-stranded RNA", NUCLEIC ACIDS RESEARCH SUPPLEMENT, no. 1, pages 219 - 220, XP002972441 *
STARCICH ET AL.: "Characterization of long terminal repeat sequences of HTLV-III", SCIENCE, vol. 227, no. 4686, January 1985 (1985-01-01), pages 538 - 540, XP002972440 *

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8957198B2 (en) 2003-02-03 2015-02-17 Medtronic, Inc. Compositions, devices and methods for treatment of Huntington's disease through intracranial delivery of sirna
US9133517B2 (en) 2005-06-28 2015-09-15 Medtronics, Inc. Methods and sequences to preferentially suppress expression of mutated huntingtin
US9273356B2 (en) 2006-05-24 2016-03-01 Medtronic, Inc. Methods and kits for linking polymorphic sequences to expanded repeat mutations
US9375440B2 (en) 2006-11-03 2016-06-28 Medtronic, Inc. Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity

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WO2003102131A2 (en) 2003-12-11
AU2003228667A1 (en) 2003-12-19

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